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About BeneFix

What is BeneFix?

BeneFix is a recombinant clotting factor IX that temporarily
replaces the factor IX that is missing or at very low levels in
people with hemophilia B.

Designed with viral safety in mind

  • Not derived from human blood and contains no preservatives or added human or
    animal components
  • Over 150 quality control tests are done on each batch of BeneFix

BeneFix is the most-prescribed recombinant
factor IX treatment for hemophilia B

Chinese hamster ovary (CHO) cells used to manufacture BeneFix are grown in media containing
no added human or animal components.

Bleed control and prevention

Demonstrated bleed control and prevention in previously untreated patients
(PUPs) and children

BeneFix prevented bleeds:

84.3%of patients

did not have spontaneous
bleeding within 48 hours of
their last infusion

Includes patients who received routine
infusions to prevent spontaneous bleeding
and intermittent administration prior to
activity or procedure.

BeneFix controlled bleeds:

75%of bleeds

in a clinical trial
were controlled after
1 infusion

  • 14.9% were controlled after 2 infusions
  • 5.4% were controlled after 3 infusions
  • 2.4% were controlled after 4 infusions
  • 2.2% were controlled after >4 infusions

STUDY DESCRIPTION FOR PREVIOUSLY UNTREATED PATIENTS

Fifty-four PUPs were administered BeneFix for on-demand treatment of any bleeding episode at a dose deemed appropriate by the
investigator, or as a prophylactic treatment to prevent either hemorrhage or surgery-related bleeding. The research was originally published
in Blood. Shapiro AD, Di Paola J, Cohen A, et al; for Recombinant Factor IX Study Group. The safety and efficacy of recombinant human
blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood. 2005;105(2):518-525.

Experience with previously treated patients (PTPs)

BeneFix prevented bleeds:

97%of bleeds

Doctors rated BeneFix as “excellent” or “good” in preventing
97% of bleeds during surgery

  • 3% of infusions were rated moderate

BeneFix controlled bleeds:

90.9%of responses

The first infusion of BeneFix was rated “excellent” or “good”
in 90.9% of responses

  • 7.1% of infusions were rated moderate
  • 0.7% of infusions were rated as no response
  • 1.3% of infusions were not rated

STUDY DESCRIPTION FOR PREVIOUSLY TREATED PATIENTS

Fifty-six PTPs were treated for bleeding episodes. The mean duration of treatment was 23.4 ± 5.3 months. The regimen was prescribed
by the investigator. The research was originally published in Blood. Roth DA, Kessler CM, Pasi KJ, Rup B, Courter SG, Tubridy KL; for
Recombinant Factor IX Study Group. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated
with plasma-derived factor IX concentrates. Blood. 2001;98(13):3600-3606.

Safety

Low levels of inhibitors in BeneFix clinical trials

The body can make antibodies called “inhibitors.” These may stop BeneFix from working properly.
The higher the level of inhibitors, the more they may block effective treatment with BeneFix.

The strength of an inhibitor in a person’s blood is measured in Bethesda units (BU).

  • High titer (more than 5 BU)
  • Low titer (less than 5 BU)

In PUPs in clinical trials:

2

out
of

63

developed a high-titer
inhibitor.

Both patients were
removed from the study.

In PTPs in clinical trials:

1

out
of

65

developed a low-titer
inhibitor.§

§This patient completed the study and had
normal factor IX recovery (about 15 months
after the inhibitor was detected).

Dosing and R2 kit

Live actively? Talk to your doctor about a BeneFix dosing
regimen that’s right for you

BeneFix delivers dosing flexibility with 5 vial sizes

  • 250 IU
  • 500 IU
  • 1000 IU
  • 2000 IU
  • 3000 IU

Flexibility and convenience:

BeneFix Rapid Reconstitution R2 Kit

  • Range of vial sizes
  • Travel-ready pack
  • Single-use vial
  • Sterile infusion set
  • Less waste

BeneFix can be stored at room temperature or under refrigeration
(2°C to 30°C/36°F to 86°F) for up to 2 years, until expiration.